NINDS Launches Clinical Trial Network, SMA Biomarkers Study

The National Institutes of Health has launched a biomarkers and natural history study in infants with type 1 spinal muscular atrophy

Article Highlights:

The National Institute for Neurological Disorders and Stroke (NINDS) is conducting a study through its new clinical trial network, Network for Excellence in Neuroscience Clinical Trials (NeuroNext). NINDS is part of the National Institutes of Health.

The study will look at natural history (general disease course) and search for disease indicators (biomarkers) in infants with type 1 spinal muscular atrophy. It will be conducted at 15 sites around the U.S., of which 12 are MDA clinics.

The new study may help researchers understand the very early stages of SMA, a necessary step in developing SMA therapies that can be administered earlier, when they may have more benefit.

"MDA is gratified that SMA was selected to be the first disease studied in the NeuroNext network," said Annie Kennedy, MDA senior vice president of advocacy. She noted that other neuromuscular diseases covered by the Association could be considered for future study by NeuroNext, adding, "We greatly appreciate NINDS' vision and leadership in this area."

The 25-site NeuroNext clinical trial network was created in 2011 to test promising new therapies for children and adults with neurological diseases and the SMA study is its first project.

The NeuroNext infrastructure involves partnerships with academia, private foundations and industry, and is designed to help maximize efficiency while minimizing the cost and time associated with conducting multicenter clinical research trials.

Study information

NINDS’s new study is designed to help researchers understand the very early stages of SMA. Such understanding is a crucial step in developing SMA therapies that could be administered during the so-called "window of opportunity" for treatment, a phrase that refers to the period from birth until the last point in time at which nerve cells (and the muscle fibers they activate) can be rescued. The exact parameters of the “window of opportunity” in SMA are unknown.

“This is a critical issue because research data indicate that there is rapid postnatal loss in SMA," said Sanjay Bidichandani, MDA vice president of research. "This study will be crucial in determining the timing and likely efficacy of future therapies for SMA.”

Study investigators plan to evaluate 54 infants, ages newborn to 6 months, whom they will assign to one of two groups: those with SMA and those without a neurological disease (the control group).